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GB202206336D0 - Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders - Google Patents

Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders

Info

Publication number
GB202206336D0
GB202206336D0 GBGB2206336.6A GB202206336A GB202206336D0 GB 202206336 D0 GB202206336 D0 GB 202206336D0 GB 202206336 A GB202206336 A GB 202206336A GB 202206336 D0 GB202206336 D0 GB 202206336D0
Authority
GB
United Kingdom
Prior art keywords
fmr1
constructs
syndrome
methods
related disorders
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
GBGB2206336.6A
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Edinburgh
Original Assignee
University of Edinburgh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Edinburgh filed Critical University of Edinburgh
Priority to GBGB2206336.6A priority Critical patent/GB202206336D0/en
Publication of GB202206336D0 publication Critical patent/GB202206336D0/en
Priority to KR1020247036991A priority patent/KR20250006355A/en
Priority to IL315634A priority patent/IL315634A/en
Priority to US18/853,310 priority patent/US20250242053A1/en
Priority to AU2023262646A priority patent/AU2023262646A1/en
Priority to CA3245707A priority patent/CA3245707A1/en
Priority to PCT/GB2023/051099 priority patent/WO2023209364A1/en
Priority to EP23725291.1A priority patent/EP4514980A1/en
Priority to JP2024553886A priority patent/JP2025514599A/en
Priority to CN202380036677.8A priority patent/CN119301258A/en
Priority to MX2024011021A priority patent/MX2024011021A/en
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Toxicology (AREA)
GBGB2206336.6A 2022-04-29 2022-04-29 Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders Ceased GB202206336D0 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
GBGB2206336.6A GB202206336D0 (en) 2022-04-29 2022-04-29 Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
CN202380036677.8A CN119301258A (en) 2022-04-29 2023-04-26 Recombinant therapeutic FMR1 constructs and methods for treating fragile X syndrome and related disorders
AU2023262646A AU2023262646A1 (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders
IL315634A IL315634A (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders
US18/853,310 US20250242053A1 (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders
KR1020247036991A KR20250006355A (en) 2022-04-29 2023-04-26 Recombinant therapeutic FMR1 constructs and methods for treating fragile X syndrome and related disorders
CA3245707A CA3245707A1 (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders
PCT/GB2023/051099 WO2023209364A1 (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders
EP23725291.1A EP4514980A1 (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders
JP2024553886A JP2025514599A (en) 2022-04-29 2023-04-26 Recombinant therapeutic fmr1 constructs and methods for treating fragile x syndrome and related disorders
MX2024011021A MX2024011021A (en) 2022-04-29 2024-09-09 Recombinant therapeutic fmr1 constructs and methods of treating fragile x syndrome and related disorders

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB2206336.6A GB202206336D0 (en) 2022-04-29 2022-04-29 Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders

Publications (1)

Publication Number Publication Date
GB202206336D0 true GB202206336D0 (en) 2022-06-15

Family

ID=81943893

Family Applications (1)

Application Number Title Priority Date Filing Date
GBGB2206336.6A Ceased GB202206336D0 (en) 2022-04-29 2022-04-29 Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders

Country Status (11)

Country Link
US (1) US20250242053A1 (en)
EP (1) EP4514980A1 (en)
JP (1) JP2025514599A (en)
KR (1) KR20250006355A (en)
CN (1) CN119301258A (en)
AU (1) AU2023262646A1 (en)
CA (1) CA3245707A1 (en)
GB (1) GB202206336D0 (en)
IL (1) IL315634A (en)
MX (1) MX2024011021A (en)
WO (1) WO2023209364A1 (en)

Family Cites Families (30)

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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
JP4108746B2 (en) 1995-06-07 2008-06-25 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル AAV production without helper virus
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP4060531B2 (en) 1998-05-28 2008-03-12 アメリカ合衆国 AAV5 vectors and uses thereof
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
DE69941905D1 (en) 1998-11-10 2010-02-25 Univ North Carolina VIRUS VECTORS AND METHOD FOR THEIR MANUFACTURE AND ADMINISTRATION.
WO2001092551A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ545628A (en) 2003-09-30 2009-04-30 Univ Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
ES2385837T3 (en) 2004-12-15 2012-08-01 The University Of North Carolina At Chapel Hill Chimeric Vectors
AU2009209408B2 (en) 2008-01-29 2015-06-11 Beacon Therapeutics Limited Recombinant virus production using mammalian cells in suspension
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
BRPI1007743A2 (en) 2009-05-12 2017-09-19 Transgene Sa IMMORTALIZED AVIAN CELL LINEAGES, USES OF AN IMMORTALIZED AVIAN CELL LINEAGE, METHODS FOR VIRUS PRODUCTION AND METHODS FOR PROTEIN PRODUCTION
CN104520421B (en) 2011-10-28 2017-05-24 北卡罗来纳-查佩尔山大学 Cell lines for the production of adeno-associated virus
DK2968605T3 (en) 2013-03-15 2022-09-26 Univ North Carolina Chapel Hill METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
BR112015021884A8 (en) 2013-03-15 2019-11-26 Childrens Hospital Philadelphia recombinant plasmid vectors, avv viral particle or plurality of viral particles, their production methods and uses, and pharmaceutical composition
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
KR102445330B1 (en) 2013-07-12 2022-09-19 더 칠드런스 호스피탈 오브 필라델피아 Assays for neutralizing antibodies to AVA vectors and anti-AVA (adeno-associated virus)
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GB2574243A (en) * 2018-05-31 2019-12-04 The Govening Council Of The Univ Of Toronto Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders
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AU2021300615A1 (en) * 2020-06-30 2023-02-02 The University Court Of The University Of Edinburgh Transgene expression system
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Also Published As

Publication number Publication date
CN119301258A (en) 2025-01-10
KR20250006355A (en) 2025-01-10
WO2023209364A1 (en) 2023-11-02
US20250242053A1 (en) 2025-07-31
AU2023262646A1 (en) 2024-09-26
EP4514980A1 (en) 2025-03-05
IL315634A (en) 2024-11-01
CA3245707A1 (en) 2023-11-02
JP2025514599A (en) 2025-05-09
MX2024011021A (en) 2024-11-08

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Legal Events

Date Code Title Description
AT Applications terminated before publication under section 16(1)